Arrakis Therapeutics is at the forefront of developing RNA-targeted small molecule (rSM) medicines, aiming to address previously "undruggable" targets associated with significant disease biology. Their innovative approach expands the potential for small molecule therapies beyond conventional methods, focusing on a diverse pipeline that includes treatments for cancer, cardiovascular diseases, neurodegeneration, and rare diseases. Notably, their lead program targets the pathological RNA involved in myotonic dystrophy type 1 (DM1), a genetic condition with no approved treatments. This program exemplifies Arrakis's commitment to creating effective therapies that can address the systemic manifestations of complex diseases.

The company employs a matrixed strategy to enhance its rSM pipeline, collaborating with major biopharmaceutical partners like Roche and Amgen to maximize the development of RNA-targeted therapies. Their research encompasses various modalities, including intrinsic function rSMs that inhibit translation of specific mRNAs, proximity degraders for targeted RNA degradation, and covalent translation inhibitors that block ribosomal progression. This multifaceted approach not only aims to create novel therapeutics but also seeks to redefine the landscape of RNA-targeted drug development, ultimately improving patient outcomes across a range of challenging health conditions.

United States

Arrakis has a simple but powerful vision: To extend small-molecule medicines into new realms of biology, unlocking that biology for medicine. Our approach builds on the existing sophisticated drug discovery toolkit developed for protein targets, which we are adapting and refocusing on discovering small-molecule compounds that act directly on RNA.

Arrakis Therapeutics

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